Invention Grant
- Patent Title: Compositions useful in treatment of ornithine transcarbamylase (OTC) deficiency
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Application No.: US15122853Application Date: 2015-03-09
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Publication No.: US10167454B2Publication Date: 2019-01-01
- Inventor: Lili Wang , James M. Wilson
- Applicant: The Trustees of the University of Pennsylvania
- Applicant Address: US PA Philadelphia
- Assignee: The Trustees of the University of Pennsylvania
- Current Assignee: The Trustees of the University of Pennsylvania
- Current Assignee Address: US PA Philadelphia
- Agency: Howson and Howson LLP
- International Application: PCT/US2015/019513 WO 20150309
- International Announcement: WO2015/138348 WO 20150917
- Main IPC: C12N15/86
- IPC: C12N15/86 ; C12N15/861 ; C12N15/867 ; C12N9/10 ; A61K48/00
Abstract:
Viral vectors comprising engineered hOTC DNA and RNA sequences are provided which when delivered to a subject in need thereof are useful for treating hyperammonemia, ornithine transcarbamylase deficiency and symptoms associated therewith. Also provided are methods of using hOTC for treatment of liver fibrosis cirrhosis in OTCD patients by administering hOTC.
Public/Granted literature
- US20170051259A1 COMPOSITIONS USEFUL IN TREATMENT OF ORNITHINE TRANSCARBAMYLASE (OTC) DEFICIENCY Public/Granted day:2017-02-23
Information query
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